Treatment Breakthrough for Advanced Melanoma

A new treatment for melanoma, the deadliest form of skin cancer, is showing promising results in patients with a particular gene mutation.

In a multi-center study just published in the New England Journal of Medicine, patients with terminal metastatic melanoma (advanced melanoma that has spread to distant organs) were treated orally with a drug called PLX4032 (a.k.a. Zelboraf), which targets a gene mutation associated with an increased risk of melanoma.

Mutations (abnormal changes) in what is known as the BRAF gene are found in 40-60 percent of all melanomas. Mutated versions of BRAF can become stuck in the "on" position, promoting uncontrolled (cancerous) growth. PLX4032/Zelboraf is a BRAF "inhibitor" that can slow or even halt this growth. In a group of 32 metastatic melanoma patients with the BRAF mutation, PLX4032/Zelboraf "induced complete or partial tumor regression [shrinking] in 81 percent of patients," according to the researchers, led by Keith T. Flaherty, MD. Responses have lasted anywhere from two to more than 18 months. It is not yet clear whether treatment with PLX4032/Zelboraf will lead to improved overall melanoma survival rates, but the authors are currently investigating this in a larger trial.